Optimalisatie van de werkingsprocessen van het Bijzonder ... - KCE

KCE Reports 133 Special Solidarity Fund 185
ask for EMEA market authorisation. The cost for the studies to be
performed is mostly too high in perspective of the number of cases
concerned. In some cases scientific studies just cannot be performed since
the patient groups are too small.
• Compassionate use is certainly no structural solution for off label use of
medication. Strictly compassionate use is limited to a one time use of the
drug and leads to inequality among patients since not all of them that are
in the same situation of a medical need, will have access to the drug. The
pharmaceutical companies ask for the introduction in Belgium of a
structural system that regulates off label use. They refer to the systems in
the surrounding countries where off label use is regulated and a
procedure for reimbursement exists.
• The medical need programs concern the use of a drug that already
obtained EMEA market authorisation but has not been accepted for
reimbursement by the compulsory health care insurance system. The
acceptance in Belgium requires a decision on acceptance of the drug and a
second one on the price of the drug.
9.13.2 Suggestions of the pharmaceutical companies
• Risk sharing system:
This system exists in the UK and in Italy. It means the public health care
insurance system will only fund the costs of the medication if the patient
reacts favourably to the treatment. At first there is no reimbursement,
later if the reaction on the treatment is positive, the drug will be
reimbursed. The difficulty in such a risk sharing system is that it is not
always clear what ‘reacting on the treatment’ implies. Criteria for
determining if the patient reacts positive to the treatment are difficult to
define. Such a system has to be transparent but may not put a too high
administrative burden on the prescribing doctor. This seems to be the
case in the UK with the consequence the system is not used as it should
be.
• Stopping rules:
Such a system allows the prescribing doctor to use the drug and see if the
patient reacts positive to the treatment. At the first stage the drug will be
reimbursed. If the patient reacts positive, the doctor has to deliver a
declaration the patient does and the treatment can be continued. If not
the treatment and the reimbursement will be terminated.
9.13.3 The view of the pharmaceutical companies on the SSF
The SSF is seen as a system that provides solutions to patients for high medical
expenses that are not covered by the compulsory health care insurance system. As such
the existence of a kind of safety net is judged as positive. The pharmaceutical sector
however formulates different remarks as to the functioning of the SSF.
Transparency of the criteria used by the SSF is poor. Even doctors and certainly patients
do not understand the criteria nor the way the SSF applies these criteria on individual
cases. SSF decisions are case by case decisions without any guarantee on later
acceptance. It would be more adequate if decisions of the SSF would be public
(anonymous) and linked to the pathology and the indications. In such a case more
certainty on acceptance would be available.The administrative burden for the
prescribing medical doctor is very high and leads to cases where no application is
introduced although theoretically the SSF could intervene.
Decisions at the SSF are taken by the College of medical directors. Since it mostly
concerns rare diseases or rare indications, one cannot expect them to have all sufficient
knowledge that is needed for an adequate judgment of each individual case. The advice
of the Drug Reimbursement Commission (DRC/CTG/CRM) is no solution since the
members of this committee do not have the expertise on innovative new drugs or on
rare indications either.